[Federal Register Volume 75, Number 83 (Friday, April 30, 2010)]
[Notices]
[Pages 22819-22821]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2010-10079]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2010-N-0218]
Considerations Regarding Food and Drug Administration Review and
Regulation of Articles for the Treatment of Rare Diseases; Public
Hearing
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public hearing; request for comment.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
hearing regarding the Agency's regulation of drugs, biological
products, and devices (e.g., therapies and diagnostics) for the
treatment, diagnosis, and/or management of rare diseases. This public
hearing is intended to gain from health care providers, academia,
industry, patients, and other interested persons their perspectives on
various aspects of the development of medical products for the
diagnosis, treatment, or management of rare diseases. The input from
this public hearing will help inform the work of FDA's committee for
rare diseases. To help solicit such information and views, FDA is
seeking responses to specific questions.
[[Page 22820]]
DATES: The public hearing will be held on June 29 and 30, 2010, from 9
a.m. to 5 p.m. However, depending on the level of public participation,
the meeting may be extended or may end early. Submit written or
electronic requests for oral presentations to Paras M. Patel (see FOR
FURTHER INFORMATION CONTACT) by May 31, 2010. Submit written comments
to the Division of Dockets Management by May 31, 2010. Submit
electronic comments to http://www.regulations.gov by May 31, 2010.
Written or electronic comments will be accepted after the hearing until
August 31, 2010.
ADDRESSES: The public hearing will be held at 10903 New Hampshire Ave.,
Bldg. 31, rm. 1503, Silver Spring, MD 20993. Additional information on
parking and public transportation may be accessed at http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm058421.htm. (FDA has verified the Web site
addresses throughout this document, but FDA is not responsible for any
subsequent changes to the Web sites after this document publishes in
the Federal Register.)
Submit written comments to the Division of Dockets Management (HFA-
305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061,
Rockville, MD 20852. Submit electronic comments to http://www.regulations.gov. Transcripts of the hearing will be available for
review at the Division of Dockets Management at http://www.regulations.gov approximately 45 days after the hearing.
FOR FURTHER INFORMATION CONTACT: Paras M. Patel, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 32, rm. 5271, Silver
Spring, MD 20993-0002, 301-796-8660, FAX: 301-847-8621, e-mail:
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
The development of therapies and diagnostics for people with rare
diseases, defined as those conditions which affect fewer than 200,000
people in the United States, presents economic and scientific
challenges. Prior to the 1983 passage of (and subsequent amendments to)
the Orphan Drug Act (ODA), the high development cost for therapies
targeting few patients was often a prohibitive economic barrier; from
1973-1982 only 12 new drugs for rare diseases were approved by FDA.
Since the ODA's passage, 357 drugs and biological products with Orphan
Designation have received FDA marketing approval. More modest advances
have been made in medical devices for people with rare diseases through
the humanitarian use device (HUD) and humanitarian device exemption
(HDE) programs. Nevertheless for most of the estimated 7,000 rare
diseases that affect an estimated 30 million Americans, no approved
therapies exist.
To optimize the means by which FDA considers articles for people
with rare diseases, a recent public law (Agriculture, Rural
Development, Food and Drug Administration, and Related Agencies
Appropriations Act, 2010, Public Law 111-80, section 740) calls for the
establishment of a committee of FDA employees to consider the means by
which the Agency reviews the data from non-clinical studies and
clinical trials, and makes decisions about marketing authorization and
postmarketing surveillance for these patient populations. This
committee, which was established March 11, 2010, is seeking public
input to benefit from a better understanding of the opinions and
suggestions of external stakeholders.
II. Purpose and Scope of the Hearing
This hearing is intended to provide advocates for patients with
rare diseases, academics, health care providers, the pharmaceutical
industry, and other interested parties an opportunity to relate their
experience with, concerns about, and suggestions for the way FDA
regulates the scientific evaluation of, marketing authorization for,
and postmarket surveillance of, articles for rare diseases. The scope
of such presentations may include non-clinical testing, clinical
trials, and decisions regarding marketing authorization and
postmarketing surveillance of products for the diagnosis or treatment
of rare diseases. FDA invites public comment from interested parties on
the following questions/issues:
1. Orphan drug marketing applications are reviewed under the same
review process and statutory standards regarding demonstration of
safety, effectiveness, and product quality as drugs for patients with
non-orphan diseases or conditions. FDA is sensitive to the unique needs
of patients with rare diseases as it makes approval decisions regarding
the overall risk-benefit profile of therapies for the particular
patient population for which they are being considered. Please comment
on whether this practice has adequately addressed the needs of patients
with rare diseases. If improvements are suggested, please provide
specific examples/suggestions for any recommended changes.
2. FDA designates a medical device as an HUD designed to treat or
diagnose a rare disease--defined in this instance as a disease
affecting or manifesting in fewer than 4,000 patients per year. Please
comment on whether this practice has adequately addressed the needs of
patients with rare diseases. Please also comment and provide your
rationale on whether 4,000 patients constitutes an appropriate
population size for an HUD determination. If improvements are
suggested, please provide specific examples/suggestions for any
recommended changes.
3. Current regulations for the approval of an HUD through the HDE
pathway require that the application have a ``description of the device
and a discussion of the scientific rationale for the use of the device
for the rare disease or condition'' and ``an explanation of why the
probable benefit to health from the use of the device outweighs the
risk of injury or illness from its use, taking into account the
probable risks and benefits of currently available devices or
alternative forms of treatment'' (21 CFR 814.102 and 814.104). Please
comment if you believe that these standards remain appropriate for the
approval of devices for rare diseases under the HDE mechanism; please
also comment whether a more precise definition of probable benefit is
needed.
4. Have current processes for rare disease stakeholders to
communicate with FDA regarding rare disease article development been
useful? How could these processes be improved? Please provide specific
examples/suggestions for any recommended changes.
III. Attendance and/or Participation in the Public Hearing
The public hearing is free and seating will be on a first-come,
first-served basis. Attendees who do not wish to make an oral
presentation do not need to register.
If you wish to make an oral presentation during the hearing, you
must register by submitting a written or electronic request by close of
business on May 31, 2010, to Paras M. Patel (see FOR FURTHER
INFORMATION CONTACT). You must provide your name, title, business
affiliation (if applicable), address, telephone and fax numbers, e-mail
address, and type of organization you represent (e.g., industry,
consumer organization). You also should submit a brief summary of the
presentation, including the discussion topic(s) that will be addressed
and the approximate time requested for your presentation. We encourage
individuals and
[[Page 22821]]
organizations with common interests to consolidate or coordinate their
presentations to allow adequate time for each request for presentation.
Persons registered to make an oral presentation should check in before
the hearing.
Participants should submit a copy of each presentation to Paras M.
Patel. We will file the hearing schedule, indicating the order of
presentation and the time allotted to each person, with the Division of
Dockets Management (see ADDRESSES). We will mail, e-mail, or fax the
schedule to each participant before the hearing. Participants are
encouraged to arrive early to ensure the designated order of
presentation.
If you need special accommodations due to a disability, please
contact Paras M. Patel at least 14 days in advance.
IV. Notice of Hearing Under 21 CFR Part 15
The Commissioner of Food and Drugs is announcing that the public
hearing will be held in accordance with part 15 (21 CFR part 15). The
hearing will be conducted by a presiding officer, accompanied by FDA
senior management from the Office of the Commissioner, the Office of
Orphan Products Development, as well as representatives from the
committee established by section 740 of the Agriculture, Rural
Development, Food and Drug Administration, and Related Agencies
Appropriations Act, 2010.
Under paragraph Sec. 15.30(f), the hearing is informal, and the
rules of evidence do not apply. No participant may interrupt the
presentation of another participant. Only the presiding officer and
panel members may question any person during or at the conclusion of
each presentation (Sec. 15.30(e)).
Public hearings under part 15 are subject to FDA's policy and
procedures for electronic media coverage of FDA's public administrative
proceedings (21 CFR 10.203(a)). Under 21 CFR 10.205, representatives of
the electronic media may be permitted, subject to certain limitations,
to videotape, film, or otherwise record FDA's public administrative
proceedings, including presentations by participants.
The hearing will be transcribed as stipulated in paragraph Sec.
15.30(b). Transcripts of the hearing will be available for review at
the Division of Dockets Management and on the Internet at http://www.regulations.gov approximately 45 days after the hearing. A
transcript will also be available in either hardcopy or on a CD-ROM
after submission of a Freedom of Information request. Submit written
requests to the Division of Freedom of Information (HFI-35), Office of
Management Programs, Food and Drug Administration, 5600 Fishers Lane,
rm. 6-30, Rockville, MD 20857.
To the extent that the conditions for the hearing, as described in
this document, conflict with any provisions set out in part 15, this
notice acts as a waiver of those provisions as specified in Sec.
15.30(h).
V. Request for Comments
Interested persons may submit written or electronic comments for
consideration to the Division of Dockets Management (see ADDRESSES).
Persons who wish to provide additional materials for consideration
should file these materials with the Division of Dockets Management.
You should annotate and organize your comments to identify the specific
questions identified by topic to which they refer. Submit a single copy
of electronic comments or two paper copies of any mailed comments,
except that individuals may submit one paper copy. Comments should be
identified with the docket number found in brackets in the heading of
this document. Received comments may be seen in the Division of Dockets
Management between 9 a.m. and 4 p.m. Monday through Friday.
Dated: April 26, 2010.
Leslie Kux,
Acting Assistant Commissioner for Policy.
[FR Doc. 2010-10079 Filed 4-29-10; 8:45 am]
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